Long Term Follow-up Study of AAVAnc80-hOTOF Gene Therapy

• Conditions: Otoferlin Gene-mediated Hearing Loss

• Registration Number: NCT06696456
• Lead Sponsor: Akouos, Inc.

Brief Summary

This multi-center, observational, post-intervention long term follow-up (LTFU) study will monitor for safety and efficacy of AAVAnc80-hOTOF in individuals with sensorineural hearing loss due to otoferlin gene mutations who have previously received intracochlear administration of AAVAnc80-hOTOF in the AK-OTOF-101 Clinical Trial.

Detailed Description

After completing one year of initial safety and efficacy assessments following an intracochlear administration of AAVAnc80-hOTOF in AK-OTOF-101, participants will continue to be observed for an additional approximately four years of safety and efficacy follow-up in this LTFU study, for a total of approximately five years of safety and efficacy observations after vector administration. For each participant, evaluations in the LTFU study will occur at approximately one and a half years and two years after vector administration, followed by annual visits through Year 5; the duration in this LTFU study is approximately four years, with the aim of understanding and mitigating any risks related to delayed adverse events, as well as characterizing the durability of effect of the AAVAnc80-hOTOF.

Study Timeline

• Study Start: 2024-11-11
• Study First Submitted: 2024-11-18
• Study First Submitted QC: 2024-11-19
• Study First Posted: 2024-11-20
• Status Verified: 2025-01
• Last Update Submitted: 2025-03-19
• Last Update Posted: 2025-03-20
• Primary Completion: 2033-04
• Study Completion: 2033-04

Recruitment & Eligibility

• Status: ENROLLING_BY_INVITATION
• Sex: All
• Target Recruitment: 30

Inclusion Criteria

• Individuals with otoferlin gene-mediated hearing loss who received intracochlear administration of AAVAnc80-hOTOF in the AK-OTOF-101 Clinical Trial
• Legally authorized representative and/or participant willingness to comply with all study requirements, as evidenced by successful completion of the informed consent process or permission and participant assent process (where appropriate)

Exclusion Criteria

• Any condition that would not allow the potential participant to complete follow-up examinations during the course of the study and/or, in the opinion of the Investigator, makes the potential participant unsuitable for the study

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Long term safety of AAVAnc80-hOTOF, including the incidence and frequency of late-occurring adverse events (AEs)	Through study completion, approximately four years.	The following adverse events will be collected: 1. The development of new or exacerbation of malignancies (oncologic), neurologic, rheumatologic or other autoimmune, hematologic disorders, or new infections (if the infection is potentially related to AAVAnc80-hOTOF; 2. AEs that are related to AAVAnc80-hOTOF and/or the administration procedure; 3. AEs of special interest; 4. All serious adverse events regardless of severity or of relationship to participation in the AK-OTOF-101 Clinical Trial

Secondary Outcome Measures

Name	Time	Method
Auditory Brainstem Response (ABR) Threshold	Through study completion, approximately four years.	Changes in ABR intensity threshold (decibels normal hearing level \[dB nHL\])

Trial Locations

Locations (3)

Children's Hospital of Philadelphia; 🇺🇸 Philadelphia, Pennsylvania, United States

Hospital Sant Joan de Déu; 🇪🇸 Esplugues de Llobregat, Barcelona, Spain

National Taiwan University Hospital; 🇨🇳 Taipei, Taiwan